Establishment of a children's Drugs and Therapeutics Committee to ensure evidence-based and cost-effective medical treatment for children.

The prevalence of undocumented medical treatments among children is a significant issue, as well as many EU countries lack access to newly developed children-friendly medicines. Consequently, there is a pressing need for supplementary resources that can facilitate informed decision-making regarding children's medication. We therefore aim to describe the process of establishing a children's Drug and Therapeutics Committee (cDTC), as well as the preparing and implementation of recommendations for children in the capital region of Denmark. Following the guidelines outlined by the World Health Organization, we established a cDTC, and recommendations for paediatric medication practice were constructed from assessments of medication use patterns among children in the capital region between 2019 and 2021. The recommendations were meticulously crafted based on evaluation of the current marketing authorization landscape and existing best available evidence. In 2019, the capital region established the first cDTC supported by expert councils and an editorial board. A total of 2429 purchase item numbers covering 1 222 846 defined daily doses and 592 088 purchased packages covering 10 200 000 defined daily doses were identified in the secondary and primary sectors, respectively. Three comprehensive lists covering recommendations for newborns and children were published between 2021 and 2020 totaling 331 recommended pharmaceutical products. The recommendations primarily intended for use in the secondary healthcare sector were implemented through the revision of 38 paediatric- and six neonatal product ranges throughout capital region. In conclusion, recommendation lists for children governed by a cDTC provide a rational auxiliary tool that can be immediately implemented in the clinic.

Massive presence of off-label medicines in Danish neonatal departments: A nationwide survey using national hospital purchase data.

There is currently insufficient knowledge of gestational age dependent medicine disposition in neonates. Accordingly, the use of off-label medication, i.e., use of medicines outside its approved marketing authorization, is high in the neonatal departments. By using data from the Danish National Pharmaceutical Hospital Purchase Database, we identified the most commonly occurring medications and calculated the on/off-label ratios for premature and term neonates. Data was extracted on ATC level 5 and based on defined daily doses as per WHO. Data covered the 4 high-level NICUs and 10 of 13 of the intermediate/standard level Danish neonatal departments. Of the identified medication, 87% and 70% did not have approved marketing authorization for use in premature and full-term neonates, respectively. Furthermore, one-fifth of the top 100 medicines did not have a (Danish) marketing license. Overall, off-label medication was widespread covering virtually all ATC groups and no ATC group had an off-label level lower than 50% (range 50%-100%). Finally, in 21% of medications, additives from 8 different chemical groups with potential deleterious effects for neonates were identified. In conclusion, off-label medication in the Danish neonatal departments is widespread. The pharmaceutical industry is unlikely to solve this problem, and we may for a very long time be occasionally forced to use off-label medication. Practical solution must therefore come from multidisciplinary clinical and academic collaboration. Use of formulation list as guidance for prescriptions and NICU-friendly galenic formulations may mitigate the problem temporarily while waiting for definitive studies.

Developing the latest framework to measure and incentivise pharmaceutical industry contributions to health research and development.

Major pharmaceutical companies contribute important expertise to health research and development (R&D), particularly in their ability to develop and bring pharmaceuticals to market. The Access to Medicine Index evaluates how 20 of the world's largest pharmaceutical companies are directing R&D efforts towards the needs of people living in low- and middle-income countries. In dissemination of its findings, the Index stimulates pharmaceutical companies to expand R&D activities in this direction. The Index methodology is reviewed every 2 years, most recently for the 2018 Index, to ensure their R&D activity is benchmarked against current health R&D priorities as defined by the global health community. The review is based on consensus-building processes involving global health stakeholders. In the latest review, two main changes to the methodology were made that will further deepen the Index's analysis of (1) how far companies' R&D activity aligns with global health priorities; and (2) whether companies make plans to ensure resulting innovations reach populations in need globally. These changes will be applied in the 2018 Access to Medicine Index. Importantly, the methodology review process highlighted the need for further prioritisation from the global health community, in particular to emphasise to innovators which product innovations are needed most critically to address the burden of non-communicable diseases in low- and middle-income countries. Should such prioritisations be developed, the Index can play an important role in tracking and stimulating company contributions towards them.